A Randomised, Double-Blind Placebo-Controlled Pilot Study Assessing the Safety and Tolerability of Arimoclomol in Adult with Sporadic Inclusion Body Myositis - Arimoclomol for Inclusion Body Myositis

Phase 1

• Conditions: The research project proposes to investigate Sporadic Inclusion Body Myositis (IBM). IBM is the commonest acquired disease of muscle affecting people aged 50 years and over. It is a progressive and debilitating disease which causes both muscle weakness and wasting, characteristically of the quadriceps and finger flexors. Over time it can lead to severe disability, falls and swallowing impairment. IBM is currently without a clinically proven, effective pharmacological treatment.; MedDRA version: 9.1 Level: LLT Classification code 10066407 Term: Inclusion body myositis

• Registration Number: EUCTR2008-008208-42-GB
• Lead Sponsor: CL - The Joint UCLH/UCL Comprehensive Biomedical Research Unit

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2009-05-29
• Study Completion: 2012-05-01
• Last Update Posted: 25 November 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 24

Inclusion Criteria

1. Meet the diagnostic criteria for definite or probable IBM (Griggs 1995)
2. Muscle function adequate for quantitative muscle testing. At least 8 of the following 16 muscle groups must have a MMT muscle grade greater or equal 3- or greater on the modified Medical Research Council Scale: Shoulder abductors, Elbow Flexors, Elbow Extensors, Wrist Flexors, Knee Extensors, Ankle Dorsiflexors, Knee Flexors, Neck Flexors (unilateral) and Neck Extensors (unilateral).
3. Age>50 years
4. Women must be post-menopausal (no menses in > 12 months) or status - post hysterectomy

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Presence of any one of the following medical conditions: diabetes mellitus or patients taking anti-diabetic medications, chronic infection, chronic renal insufficiency, cancer other than skin cancer less than 5 years previously, multiple sclerosis or prior episode of central nervous system demyelination, or other chronic serious medical illnesses
2. Presence of any of the following on routine blood screening: WBC<3000/cm3, platelets<100,000/cm3, hematocrit < 30 %, urea > 10 millimoles/litre, creatinne > 150 micromoles/litre, symptomatic liver disease with serum albumin < 30g/L, prothrombin time or activated partial thromboplastin time > upper range of control values
3. Currently taking Riluzole
4. Women who are pregnant or lactating
5. History of non-compliance with other therapies
6. Coexistence of other neuromuscular disease
7. Drug or alcohol abuse within last 3 months
8. Inability to give informed consent
9. Known bleeding disorder
10. Use of potentially renal-toxic drugs
11. Prior difficulties with local anesthetic

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified